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Rustum Karanjia, Stuart G Coupland, Martin Garcia, Alfredo A Sadun; Elamipretide (MTP-131) Topical Ophthalmic Solution for the Treatment of Leber's Hereditary Optic Neuropathy. Invest. Ophthalmol. Vis. Sci. 2019;60(9):2266.
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Leber’s Hereditary Optic Neuropathy is a mitochondrial disease characterized by sudden and profound vision loss in both eyes. Elamipretide has been demonstrated to colocalize with cardiolipin in the inner membrane of mitochondria, where it is thought to improve mitochondrial bioenergetics. The purpose of this study was to look at the safety, tolerability and potential efficacy of topical Elamipretide in patients affected with LHON.
Twelve patients affected with LHON were included in this study. Patients between ages 18 and 50 with decreased vision, for at least 1 year and no more than 10 years, and a genetically confirmed diagnosis of 11778 LHON were eligible for this trial. Patients who had concomitant medical conditions were excluded from the study. The primary outcome measure was the assessment of adverse events from the administration of topical Elamipretide 1%. Secondary outcome measures looked at changes in patient’s visual acuity, color vision, visual field mean deviation and electrophysiological outcomes. Patients were randomized to one of two arms, Elamipretide in both eyes or Elamipretide in one eye and vehicle in the other eye.
Seventeen patients were screened for this study to find 12 patients who met the inclusion criteria. There were no serious adverse events reported during the first 52 weeks of the study. There were 9 cases of mild to moderate ocular irritation in patients who were using Elamipretide. There was one severe ocular event in a subject who developed choroidal neovascularization secondary to trauma, unrelated to the use of the medication.
Elamipretide topical solution is safe for ocular administration with no serious adverse events reported and mild to moderate ocular irritation as the main effect. Analysis of the efficacy data is ongoing. All 12 patients have continued in an open label extension study where both eyes are being treated with Elamipretide.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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